A Method of Delivering Genes to the Central Nervous System

Inventor(s)

Tech ID

FN9132

Background

One of the limitations of gene therapy has been the lack of methods to effectively deliver genes to the central nervous system. Herpes simplex virus (HSV) is a neurotropic virus that naturally establishes latent infections in the peripheral nervous system and the central nervous system of humans. Wistar researchers have developed a novel method for using neurotropic HSV to deliver genes of therapeutic value. Additionally, the Wistar technology promotes long-term expression of the therapeutic genes, which is necessary for treatment of a genetic defect.

The Wistar technology has several advantages over other methods of delivering genes to the central nervous system (CNS). First, no helper virus is needed, and second, long-term expression of the gene is achieved using a naturally-occurring viral promoter. This technology has been used successfully in vivo in a mouse model. In this study, the gene encoding §-glucorinodase was delivered to the CNS and long-term expression (over 4 months) of the gene product was obtained.

Key Words

central nervous system, gene therapy, Therapeutic

Applications and Advantages

This technology can be used to treat a variety of genetic diseases that affect the CNS, such as lysosomal storage diseases (e.g. Tay-Sachs, Neimann-Pick, Sly's Disease). The technology may also be useful for treating other conditions that result from a biochemical deficiency in the CNS (e.g. ParkinsonÕs Disease, possibly AlzheimerÕs), or in which delivery of other gene products may be useful (e.g. delivery of cytokines).